Study: Price, therapeutic value impact international submission times for new drugs

The US tends to receive regulatory submissions earlier and more often than other major international regulators, but there was a similar rate of submissions across regulators for oncology drugs and those deemed by authorities of having moderate-to-high therapeutic value, according to recent research published in Health Affairs.

While the US Food and Drug Administration received a majority of new drug submissions, the agency also received a higher proportion of non-cancer, non-orphan drugs, drugs not on expedited approval pathways, and drugs considered by regulators as having a low therapeutic value when compared to other international regulators, Irene Papanicolas, a health economist and health services researcher at Brown University in Providence, Rhode Island, and colleagues said. However, when oncology drugs and moderate-to-high therapeutic value drugs were compared, the submission times were similar across international regulators, they noted.

The researchers compared 241 drugs approved by either the FDA or the European Medicines Agency (EMA) between 2014 and 2018 and then approved by FDA, EMA, Health Canada, Pharmaceuticals and Medical Devices Agency (PMDA) in Japan, or the Therapeutic Goods Administration (TGA) in Australia through 2022. Papanicolas and colleagues analyzed the submission times for the drugs at each regulator, and compared submissions times based on therapeutic area, therapeutic value, orphan status, whether the drug was part of an expedited pathway, launch price, and market size.

Of the 241 drugs approved between 2014 and 2018, 237 drugs (98%) were approved by FDA and 206 drugs (85%) by EMA through 2022 compared to Health Canada (71%), TGA (63%), and PMDA (49%). Overall, 70% of new drugs were submitted to FDA first, 27% to EMA first, 7% to PMDA, and 1% to Health Canada, with no drug submissions to TGA.

Researchers found the median delay from first submission was 0 months for FDA, 0.6 months for EMA, 7.9 months for Health Canada, 9.6 months for PMDA, and 18.5 months for TGA. While FDA had the same rate of submission for cancer drugs when compared to non-cancer drugs, cancer drugs were more likely to be submitted at EMA (93% vs 83%), Health Canada (90% vs 65%), PDMA (61% vs 46%), and TGA (83% vs 56%) compared to non-cancer drugs. Submission rates were higher for drugs considered by at least one regulator to be of moderate-to-high therapeutic value than low therapeutic value at EMA (98% vs 81%), Health Canada (94% vs 63%), PDMA (73% vs 41%), and TGA (87% vs 54%).

The median regulatory review time was 9.6 months for FDA compared to 9.2 months at the PDMA, 11.4 months at Health Canada, 11.6 months at the TGA, and 14.1 months at the EMA. However, median regulatory review times across international agencies were shorter for cancer drugs, drugs with moderate-to-high therapeutic value, drugs granted accelerated approval by FDA, and orphan drugs, with EMA having the same median regulatory review time for both orphan and non-orphan drugs. Following FDA approval, the median time to marketing authorization was 5.2 months for EMA, 8.4 months for PDMA, 9.6 months for Health Canada, and 11.9 months for the TGA.

Factors that influenced submission delays included whether a drug had an orphan drug status, its therapeutic value, launch price, and the drug’s market size, the researchers said. On average, submission for orphan drugs were delayed by 4 months when compared to non-orphan drugs, drug submissions were an average of 6 months earlier for those considered to be of moderate-to-high value compared to those of low therapeutic value.

“These findings have important implications for policy makers in the US and elsewhere who are interested in understanding the factors that may influence the availability of novel therapeutics for their populations,” Papanicolas and colleagues wrote.

They highlighted the difference in the processes for setting product prices in the US and the other countries studied, noting that brand-name manufacturers in the US can set product prices at launch, while in other countries, there is typically a price negotiation that is based on the effectiveness or cost-effectiveness of new drug from a health technology assessment body.

“These mechanisms may influence the decision of companies regarding whether and when to submit drugs with low therapeutic value to other regulators,” the researchers concluded.

Health Affairs Papanicolas et al.

Study: Price, therapeutic value impact international submission times for new drugs

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